Medical options for bleeding disorders

Oral contraceptives (The Pill) raise the levels of factors II, VII, VIII, X and von Willebrand factor in the blood. For many women with bleeding disorders and who suffer from menorrhagia, this hormone therapy alone is effective in reducing bleeding to normal.

This hormone therapy will not improve factor levels for women with:

• deficiencies in factors I, V, IX, XI and XIII and
• Type 2 VWD (which is not a problem of quantity of VWF but rather the way it works)

However, oral contraceptives can be helpful even for these women. They regulate the menstrual periods and reduce the flow of blood.

Other hormone therapies may be prescribed when oral contraceptives do not work well. These include progesterone. They work by thickening the lining of the uterus. This makes it less prone to bleed. However, these products can not be taken for long periods of time.

Desmopressin is a synthetic drug which is a copy of a natural hormone. It acts by releasing VWF stored in the lining of the blood vessels. The increased VWF in turn transports extra factor VIII. Desmopressin is not made from blood.

Desmopressin is the treatment of choice for Type 1 VWD, hemophilia A carriers and most platelet function disorders. It is sometimes effective in Type 2A VWD.

Desmopressin can be taken in three different ways.

• It can be injected into a vein. Most often, the brand name for this kind of desmopressin is DDAVP.
• It can be injected under the skin. The brand name for this kind of desmopressin is often Octostim.
• It can be taken by nasal spray. The brand name of the nasal spray is often Octostim Spray.

Desmopressin is effective for almost all people with Type 1 VWD. However, different people respond to desmopressin in different ways. Therefore, a doctor needs to do tests to find out each individual's response to the drug. Ideally, these tests are done before any urgent need for the drug, such as surgery.

Since desmopressin acts by releasing VWF stored in the body, you cannot 'go to the well' too often. A sufficient amount of time, usually 24 hours, must elapse between doses of desmopressin to allow the body to rebuild its stores.

In major surgery, desmopressin alone may not be enough to control bleeding. In such a case, a person should also receive factor concentrates. (See Clotting factor concentrates.)

Desmopressin is of no help to women with:

Desmopressin can sometimes have some mild side effects. These are

If a person has a very bad headache or has not been able to pass water 24 hours after taking desmopressin, he / she should go to the Hemophilia / Bleeding Disorder Treatment Centre or emergency room for help.

Cyklokapron (tranexamic acid) and Amicar (aminocaproic acid) are drugs that help to hold a clot in place once it has formed. They act by stopping the activity of an enzyme, called plasmin, which dissolves blood clots.

They do not help to actually form a clot. This means they can not be used instead of desmopressin, VWF concentrate or factor concentrates.

They can be used to hold a clot in place in mucous membranes such as:

• the inside of the mouth
• the inside of the nose
• inside the intestines (the gut)
• inside the uterus (the womb).

Cyklokapron and Amicar have proven very useful for women with bleeding disorders. They are used:

For women with menorrhagia, Cyklokapron and Amicar can be started on the first day of menstrual bleeding and taken for 5 days in a row. They can even be combined with the use of oral contraceptives for women who do not respond to desmopressin.

Cyklokapron and Amicar come in tablet form. There is even a popsicle form of Amicar for controlling bleeding in the mouth in children.

Cyklokapron and Amicar can sometimes have some mild side effects. These are:

These mild side effects go away when:

• when desmopressin, hormone therapy and anti-fibrinolytic drugs are not effective
• for surgery or
• after serious accidental injury.

Most factor concentrates are made from pooled human plasma. Plasma is a yellow liquid contained in blood. The plasma has been fractionated to take out the needed factors. This means that the different parts of the blood have been separated from each other so each person receives only that part which he / she needs.

The plasma used to make concentrates is screened for blood-borne viruses such as HIV and hepatitis B and C. Any plasma found to contain these viruses is not used. The screened plasma is then pasteurized to destroy any remaining viruses. Factor concentrates used today have an excellent safety record.

However, they may transmit parvovirus. Parvovirus is a common virus which is not normally dangerous. However, it can cause miscarriage (spontaneous abortion). Therefore, pregnant women, or women who might become pregnant, should avoid factor concentrates made from plasma, if possible.

Three concentrates( marked with an asterisk in the list above) - factors VIIa, VIII and IX - are genetically-engineered, recombinant (artificial) products. This means that they are not made from human plasma. They do not transmit parvovirus. As a result, these concentrates are the treatments of choice for factor VII deficiency and for hemophilia A and B carriers.

Factor concentrates are injected into a vein. They can be administered at a clinic, doctor's office or emergency room. Many people learn to inject them at home. (See Clotting factor concentrates.)

* Oral contraceptives, other hormone therapy, thrombin and antifibrinolytic drugs may also be useful in particular circumstances.